On track to report topline data mid-2020
Albireo is on track to report topline results in mid-2020. Albireo expects results from a second NASH/NAFLD trial with elobixibat, sponsored by its Japanese partner EA Pharma, late this year or early next year. These two proof-of-concept studies are designed to assess the combination of improvements in liver function and cardiovascular risk parameters with a favorable gastrointestinal tolerability profile. The combined data from both studies will inform next steps for Albireo.
“This is the first clinical trial ever conducted in both NASH and NAFLD patients with an IBAT inhibitor,” said
The proof-of-concept Phase 2, multicenter, placebo-controlled trial enrolled 47 patients, and is designed to assess the safety and efficacy of a once-daily 5mg dose of elobixibat over 16 weeks in adult patients across 10 U.S. sites with biopsy-confirmed NASH, or a diagnosis of suspected NASH or NAFLD based on metabolic syndrome definitions. The primary endpoint is change from baseline in serum low-density lipoprotein cholesterol (LDL-C). Secondary endpoints include change in liver fat by imaging, and in alanine transaminase (ALT) and aspartate aminotransferase (AST). Exploratory endpoints include measures of glucose and insulin homeostasis, biomarkers for inflammation and fibrosis.
In addition, EA Pharma, the exclusive licensee of elobixibat for the treatment of GI disorders in
Albireo’s adult liver disease program also includes a lead product candidate that is a novel investigational preclinical bile acid modulator, for which the company expects to complete IND-enabling studies this year.
“We believe that, if successful, our approach to NASH/NAFLD could offer an effective and novel therapeutic approach as either monotherapy or combination therapy with a favorable safety profile in an area of massive unmet need,” Cooper said. “While we remain focused on advancing odevixibat in progressive familial intrahepatic cholestasis (PFIC) and other rare pediatric cholestatic liver diseases, our NASH/NAFLD program has potentially strong value for partnering opportunities.”
NAFLD affects about 25 percent of the world’s population and is a major cause of liver disease. NAFLD may progress to NASH, a common, serious and sometimes fatal chronic liver disease characterized by liver inflammation and damage caused by a buildup of fat in the liver. NASH patients have a 10-fold greater risk of liver-related mortality, compared with the general population, and NASH is projected to become the leading cause of liver transplants in
Elobixibat is a first-in-class, once-daily, orally-available ileal bile acid transporter (IBAT) inhibitor currently being evaluated in a Phase 2 clinical trial in nonalcoholic fatty liver disease (NAFLD) and nonalcoholic steatohepatitis (NASH). In clinical studies, elobixibat demonstrated a decrease in LDL/H cholesterol, as well as decreased insulin resistance through an increase in GLP-1. The first IBAT inhibitor approved globally, elobixibat is approved in
Albireo was spun out from AstraZeneca in 2008.
This press release includes “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include statements, other than statements of historical fact, regarding, among other things: the plans for, or progress, scope, cost, initiation, duration, enrollment, results or timing for availability of results of, development of odevixibat, elobixibat or any other Albireo product candidate or program, including regarding the Phase 3 clinical program for odevixibat in patients with PFIC; the Phase 2 clinical trial for elobixibat in NAFLD/NASH, the target indication(s) for development, the size, design, population, location, conduct, objective, enrollment, duration or endpoints of any clinical trial, or the timing for initiation or completion of or reporting of results from any clinical trial, including the double-blind Phase 3 PFIC trial for odevixibat, and the long-term open-label extension study, or the Phase 2 trial for elobixibat in NAFLD/NASH; the potential approval and commercialization of odevixibat; discussions with the FDA regarding our programs; the potential benefits or competitive position of odevixibat, elobixibat, or any other Albireo product candidate or program or the commercial opportunity in any target indication; the potential benefits of an orphan drug designation; the pricing of odevixibat if approved; or Albireo’s plans, expectations or future operations, financial position, revenues, costs or expenses. Albireo often uses words such as “anticipates,” “believes,” “plans,” “expects,” “projects,” “future,” “intends,” “may,” “will,” “should,” “could,” “estimates,” “predicts,” “potential,” “planned,” “continue,” “guidance,” and similar expressions to identify forward-looking statements. Actual results, performance or experience may differ materially from those expressed or implied by any forward-looking statement as a result of various risks, uncertainties and other factors, including, but not limited to: whether favorable findings from clinical trials of odevixibat to date, including findings in indications other than PFIC, will be predictive of results from the trials comprising the Phase 3 PFIC program or any other clinical trials of odevixibat; whether either or both of the FDA and EMA will determine that the primary endpoint for their respective evaluations and treatment duration of the double-blind Phase 3 trial in patients with PFIC are sufficient, even if the primary endpoint is met with statistical significance, to support approval of odevixibat in the United States or the European Union, to treat PFIC, a symptom of PFIC, a specific PFIC subtype(s) or otherwise; the outcome and interpretation by regulatory authorities of the ongoing third-party study pooling and analyzing of long-term PFIC patient data; the timing for initiation or completion of, or for availability of data from, clinical trials of odevixibat, including the trials comprising the Phase 3 PFIC program; Albireo’s ability to obtain coverage, pricing or reimbursement for approved products in the United States or European Union; delays or other challenges in the recruitment of patients for, or the conduct of, the double-blind Phase 3 trial or other pivotal trials; and Albireo’s critical accounting policies. These and other risks and uncertainties that Albireo faces are described in greater detail under the heading “Risk Factors” in Albireo’s most recent Annual Report on Form 10-K or in subsequent filings that it makes with the Securities and Exchange Commission. As a result of risks and uncertainties that Albireo faces, the results or events indicated by any forward-looking statement may not occur. Albireo cautions you not to place undue reliance on any forward-looking statement. In addition, any forward-looking statement in this press release represents Albireo’s views only as of the date of this press release and should not be relied upon as representing its views as of any subsequent date. Albireo disclaims any obligation to update any forward-looking statement, except as required by applicable law.
Source: Albireo Pharma, Inc.